RPE65 and encephalopathy, progressive, early-onset, with brain edema and/or leukoencephalopathy: Four applications (20%)– triheptanoin (approved for a source of calories and fatty acids in the treatment of long-chain fatty acid oxidation disorders [LC-FAOD]) [29], stiripentol (for Dravet syndrome) [34], voretigene neparvovec (for biallelic RPE65 mutation-associated retinal dystrophy) [38], and vestronidase alfa-vjbk (for MPS VII) [41]– utilized retrospective medical chart reviews for RWD.